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Palpitations Over a New Pill for Kids

Cholesterol-lowering statin drugs, the most prescribed pills in the world, have become a family affair.

Middle-aged men, many women and the elderly are routinely put on the powerful drugs to lower high cholesterol. Now the nation’s leading pediatric group says that certain high-risk children as young as 8 may also be put on statin therapy, just like Mom, Dad and Grandpa.

The new guidelines from the American Academy of Pediatrics have been sharply criticized by many pediatricians and parents. They worry about the long-term health consequences of the drugs and have raised questions about financial ties between the academy and drug companies. There is also concern that the guidelines could lead to more widespread use of the drugs among children. An estimated 13 percent of children have total cholesterol above 200 milligrams per deciliter, the threshold used in adults to determine high cholesterol.

But the doctors who wrote the guidelines say they have been largely misunderstood. They say that far from leading to widespread use of statin drugs by children, the guidelines target the small percentage of children with genetic cholesterol problems or those with several worrisome risk factors, like obesity, high blood pressure and diabetes.

“I don’t see this as a major groundswell for the indiscriminate use of lipid-lowering drugs,” said Dr. Stephen Daniels, a member of the A.A.P.’s nutrition committee and chairman of the department of pediatrics at the University of Colorado Denver School of Medicine. “That’s exactly why we need these guidelines, to say where the limits of that usage should be.”

It’s not clear how many children will be affected. About 1 in 500 have genetic cholesterol disorders, and doctors estimate that currently, about 30 percent to 60 percent of those children haven’t had a diagnosis. Over all, only about 5 percent of children have levels of LDL, or “bad” cholesterol, above 130. Drug therapy would be suggested for children with bad cholesterol of 130 only if they have diabetes. Otherwise, drugs would be recommended for those with bad cholesterol of 160 and a family history of heart disease or two other risk factors, or when bad cholesterol hits 190.

Already five statin drugs — Bristol-Myers Squibb’s Pravachol, Pfizer’s Lipitor, Merck’s Zocor and Mevacor, and Novartis’s Lescol — have been approved for use in children with genetic cholesterol disorders. But there is little data on long-term use.

“People should realize that these drugs have only been studied in children with a genetic defect that causes very high cholesterol levels,” said Dr. Dianne Murphy, director of the Food and Drug Administration’s office of pediatric therapeutics, who noted that those children were followed for only two years.

Some doctors say the short-term data on statin use in children is reassuring and mirrors long-term safety data in adults. “The concept is that prevention should start early,” said Dr. Daniels. “You already have children who have risk factor levels that would be a concern for an adult.”

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But the lack of long-term data has caused concern among many pediatricians, who say children often metabolize drugs differently than adults.

Dr. Darshak Sanghavi, a pediatric cardiologist at the University of Massachusetts Medical School, said statin drugs may affect a child’s endocrine system, which regulates growth and development, among other things. “I, for one, feel unsafe simply saying children are little adults in this case,” he said.

The medical literature is filled with examples of mistakes made when medical experts extrapolated data from one group to another. For years, doctors assumed that since menopause hormones appeared to protect the hearts of middle-aged women, then older women would benefit even more. But when the issue was studied in the large Women’s Health Initiative trial, older women on hormones turned out to have a far higher risk for heart attack and stroke.

The new guidelines have raised questions about the pharmaceutical industry’s ties to both the A.A.P. and the members of the group’s nutrition committee that made the recommendations. But the A.A.P. asserted last week that “there is no involvement by any commercial entity in the development of any statement or report” it issues.

Reports filed with the Internal Revenue Service and provided by the A.A.P. show that the academy has received contributions from several companies with ties to statins, including $433,000 from Merck, $835,250 from Abbott Laboratories’ Ross Product Division and $216,000 from the Bristol-Myers Squibb company Mead Johnson Nutritionals. The biggest statin maker, Pfizer, is not listed as a contributor. The A.A.P. reported a total of $81 million in revenue in 2007.

Three committee members have disclosed industry ties. Dr. Daniels was a one-time consultant for Merck and has also worked for Abbott Laboratories, although not on cholesterol drugs. Dr. Nicolas Stettler of the Children’s Hospital of Philadelphia took part in clinical trials for Merck’s Mevacor and a failed Pfizer cholesterol drug. Dr. Jatinder Bhatia, chief of neonatology at the Medical College of Georgia in Augusta, couldn’t be reached for comment, but in earlier disclosure statements he listed financial relationships with Bristol-Myers Squibb’s Mead Johnson unit, Abbott’s Ross Products and Dey Laboratories, which is affiliated with Merck.

Two committee members — Dr. Frank Greer, the committee chairman and a neonatologist at the University of Wisconsin, Madison; and Dr. Marcie Schneider, a pediatrician in Norwich, Conn. — said they had no financial relationships with the drug industry. Two others could not be reached for comment.

But the notion that the A.A.P. recommendations will turn into a bonanza for the drug industry — at least in the short term — is unlikely. Because five statins have already been approved for pediatric use, and most of the drugs have already gone off patent or are nearing the end of their patents, additional growth opportunities for the existing branded drugs are limited.

Regardless, many in the pediatrics community appear ready to resist the notion of putting children on statin therapy. Dr. Barney Softness, associate professor of pediatric endocrinology at Columbia University’s College of Physicians and Surgeons, who regularly treats diabetic children, said he would be reluctant to prescribe a statin to a child. He notes that he himself stopped taking the statin Lipitor because of muscle aches.

“There’s no data yet on long-term safety and efficacy studies in children,” Dr. Softness said. “I just don’t think the drug is benign enough to take the chance on some long-term side effects.”

A version of this article appears in print on , on Page WK3 of the New York edition with the headline: Palpitations Over A New Pill for Kids. Order Reprints|Today's Paper|Subscribe